-Statistically significant improvements in weight gain and in
patient-reported quality of life as measured by the respiratory domain
of the Cystic Fibrosis Questionnaire Revised (CFQ-R)-

-Supplemental New Drug Application (sNDA) in the United States and
Marketing Authorization Application (MAA) variation in Europe planned
for second half of 2013 for gating mutations-

CAMBRIDGE, Mass.--(BUSINESS WIRE)--
Vertex
Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the
first clinical data from one of the company's ongoing Phase 3
label-expansion studies for ivacaftor monotherapy in people with cystic
fibrosis (CF). In the Phase 3 study evaluating ivacaftor monotherapy in
people ages six and older with at least one non-G551D gating mutation,
the mean absolute treatment difference in percent predicted FEV1
between treatment with ivacaftor and placebo was 10.7% (p < 0.0001) and
the mean relative treatment difference in percent predicted FEV1
was 14.2% (p < 0.0001) through the 8-week treatment period. KALYDECO
(ivacaftor) is currently approved for people with cystic fibrosis (CF)
ages 6 and older who have at least one copy of the G551D mutation in the
cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Worldwide, approximately 2,000 people with CF ages six and older have at
least one copy of the G551D mutation, and approximately 400 people with
CF ages six and older have at least one non-G551D gating mutation. The
study in gating mutations is one of three ongoing Phase 3
label-expansion studies for ivacaftor designed to evaluate whether
additional people with CF may benefit from treatment with ivacaftor.

Based on these data, Vertex plans to submit a supplemental New Drug
Application (sNDA) in the United States and a Marketing Authorization
Application (MAA) variation in Europe in the second half of 2013 for the
use of ivacaftor monotherapy in people with CF ages 6 and older who have
at least one non-G551D CFTR gating mutation.

"Our goal in CF is to help as many people as possible with our
medicines, and these data are an important step toward that goal," said
Robert Kauffman, M.D., Ph.D., Senior Vice President and Chief Medical
Officer at Vertex. "The data announced today in people with CF who have
a gating mutation showed a meaningful improvement in lung function and
support our plans to seek approval of ivacaftor for these patients later
this year."

About the Study

The Phase 3 crossover study of ivacaftor enrolled 39 people with CF ages
6 and older who have at least one non-G551D gating mutation. Patients
received either ivacaftor, or placebo, for eight weeks, followed by a
4-week washout period. Following the washout period, patients who
received placebo in the first eight weeks received ivacaftor for weeks
12 to 20, and patients who received ivacaftor for the first eight weeks
received placebo for weeks 12 to 20. The primary analysis was conducted
at week 20 of the study, and the primary endpoint of the study was
absolute change from baseline in percent predicted FEV1.
Following the end of the crossover periods, all patients receive
ivacaftor from week 20 to week 36 as part of an open-label dosing period.

In this study, the mean absolute treatment difference in percent
predicted FEV1 between treatment with ivacaftor and placebo
was 10.7% (p < 0.0001) and the mean relative treatment difference in
percent predicted FEV1 was 14.2% (p < 0.0001) through the
8-week treatment period. The study met its primary endpoint. The mean
absolute and relative percent predicted FEV1 improvements
during ivacaftor treatment (within-group) were 7.5% (p < 0.0001) and 10.8%
(p < 0.0001), respectively. Additionally, treatment with ivacaftor in this
study resulted in statistically significant improvements in weight gain
and in improvements in patient-reported quality of life as measured by
the respiratory domain of the Cystic Fibrosis Questionnaire Revised
(CFQ-R). Full data from this study will be submitted for presentation at
a medical meeting in the second half of 2013.

The safety and tolerability results observed in this study were
consistent with those observed in prior Phase 3 studies of ivacaftor
monotherapy in people with CF who have the G551D mutation. The most
commonly observed adverse events, regardless of treatment assignment,
included pulmonary exacerbation, cough, headache and abdominal pain,
each occurring more frequently while patients received placebo than when
patients received ivacaftor.

Two additional Phase 3 label-expansion studies are ongoing for ivacaftor
monotherapy, including a study in people with CF ages 6 and older who
have at least one copy of the R117H mutation and a study in children
with CF ages 2 to 5 who have a gating mutation, including the G551D
mutation. A Phase 2 proof-of-concept study evaluating ivacaftor in
people with CF who have clinical evidence of residual CFTR function is
also ongoing.

Indication and Important Safety Information for KALYDECOTM
(ivacaftor)

Ivacaftor (150mg tablets) is indicated for the treatment of cystic
fibrosis (CF) in patients age 6 years and older who have a G551D
mutation in the CFTR gene.

Ivacaftor is not for use in people with CF due to other mutations in the CFTR gene.
It is not effective in CF patients with two copies of the F508del
mutation (F508del/F508del) in the CFTR gene. The
efficacy and safety of ivacaftor in children younger than 6 years of age
have not been evaluated.

High liver enzymes (transaminases, ALT and AST) have been reported in
patients receiving ivacaftor. It is recommended that ALT and AST be
assessed prior to initiating ivacaftor, every 3 months during the first
year of treatment, and annually thereafter. Patients who develop
increased transaminase levels should be closely monitored until the
abnormalities resolve. Dosing should be interrupted in patients with ALT
or AST of greater than 5 times the upper limit of normal. Following
resolution of transaminase elevations, consider the benefits and risks
of resuming ivacaftor dosing. Moderate transaminase elevations are
common in subjects with CF. Overall, the incidence and clinical features
of transaminase elevations in clinical trials was similar between
subjects in the ivacaftor and placebo treatment groups. In the subset of
patients with a medical history of elevated transaminases, increased ALT
or AST have been reported more frequently in patients receiving
ivacaftor compared to placebo.

Use of ivacaftor with medicines that are strong CYP3A inducers such as
the antibiotics rifampin and rifabutin; seizure medications
(phenobarbital, carbamazepine, or phenytoin); and the herbal supplement
St. John's Wort substantially decreases exposure of ivacaftor, which may
diminish effectiveness. Therefore, co-administration is not recommended.

The dose of ivacaftor must be adjusted when concomitantly used with
potent and moderate CYP3A inhibitors. The dose of ivacaftor must be
adjusted when used in patients with moderate or severe hepatic disease.

Ivacaftor can cause serious adverse reactions including abdominal pain
and high liver enzymes in the blood. The most common side effects
associated with ivacaftor include headache; upper respiratory tract
infection (the common cold), including sore throat, nasal or sinus
congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash;
and dizziness. These are not all the possible side effects of ivacaftor.
A list of the adverse reactions can be found in the full product
labeling for each country where ivacaftor is approved. Patients should
tell their healthcare providers about any side effect that bothers them
or doesn't go away.

Vertex creates new possibilities in medicine. Our team discovers,
develops and commercializes innovative therapies so people with serious
diseases can lead better lives.

Vertex scientists and our collaborators are working on new medicines to
cure or significantly advance the treatment of hepatitis C, cystic
fibrosis, rheumatoid arthritis and other life-threatening diseases.

Founded more than 20 years ago in Cambridge, Mass., we now have ongoing
worldwide research programs and sites in the U.S., U.K. and Canada.
Today, Vertex has more than 2,000 employees around the world, and for
three years in a row, Science magazine has named Vertex one of
its Top Employers in the life sciences.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements as defined in the
Private Securities Litigation Reform Act of 1995, including, without
limitation, Dr. Kauffman's statements in the third paragraph of this
press release and statements regarding (i) Vertex's plan to submit an
sNDA in the United States and an MAA variation in Europe in the second
half of 2013 and (ii) Vertex's plan to submit full data from this study
for presentation at a medical meeting in the second half of 2013. While
Vertex believes the forward-looking statements contained in this press
release are accurate, there are a number of factors that could cause
actual events or results to differ materially from those indicated by
such forward-looking statements. Those risks and uncertainties include,
among other things, that Vertex could experience unforeseen delays in
submitting regulatory filings, and other risks listed under Risk Factors
in Vertex's annual report and quarterly reports filed with the
Securities and Exchange Commission and available through the company's
website at www.vrtx.com.
Vertex disclaims any obligation to update the information contained in
this press release as new information becomes available.